A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)


Study First Submitted Date 2020-08-25
Study First Posted Date 2020-08-28
Last Update Posted Date 2021-10-20
Verification Month Year October 2021
Verification Date 2021-10-31
Last Update Posted Date 2021-10-20

Detailed Descriptions

Sequence: 20753032
Description The objective of this EAP is to provide access to maralixibat for eligible ALGS patients with cholestatic pruritus. Participants will be treated with 400 µg/kg/day with safety and tolerability evaluated on an ongoing basis.


Sequence: 200369962 Sequence: 200369963 Sequence: 200369964 Sequence: 200369965 Sequence: 200369966 Sequence: 200369967 Sequence: 200369968 Sequence: 200369969 Sequence: 200369970 Sequence: 200369971 Sequence: 200369972 Sequence: 200369973 Sequence: 200369974 Sequence: 200369975
Name University of California, Los Angeles Name Stanford University Name University of California San Francisco Name Georgetown University Name Advent Health Name Children’s Healthcare of Atlanta/Emory University Name Ann & Robert H. Lurie Children’s Hospital Name Ochsner Clinic Foundation Name University of Minnesota Name The Children’s Hospital at Montefiore Name University of Oklahoma Name Baylor College of Medicine Name University of Virginia Name Medical College of Wisconsin
City Los Angeles City Redwood City City San Francisco City Washington City Orlando City Atlanta City Chicago City New Orleans City Minneapolis City New York City Oklahoma City City Houston City Charlottesville City Milwaukee
State California State California State California State District of Columbia State Florida State Georgia State Illinois State Louisiana State Minnesota State New York State Oklahoma State Texas State Virginia State Wisconsin
Zip 90095 Zip 94063 Zip 94158 Zip 20007 Zip 32804 Zip 30329 Zip 60611 Zip 70121 Zip 55455 Zip 10467 Zip 73104 Zip 77030 Zip 22903 Zip 53226
Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States Country United States


Sequence: 52251354
Name Alagille Syndrome
Downcase Name alagille syndrome

Id Information

Sequence: 40217127
Id Source org_study_id
Id Value MRX-EAP


Sequence: 42632048
Name United States
Removed False


Sequence: 52564284
Intervention Type Drug
Name maralixibat
Description Maralixibat is an Apical Sodium-dependent Bile acid Transporter (ASBT) inhibitor


Sequence: 79982414 Sequence: 79982415 Sequence: 79982416 Sequence: 79982417 Sequence: 79982418 Sequence: 79982419 Sequence: 79982420 Sequence: 79982421
Name Liver Disease Name ALGS Name Maralixibat Name Bile Duct Diseases Name Digestive System Diseases Name Biliary Tract Diseases Name Cholestasis Name Pruritis
Downcase Name liver disease Downcase Name algs Downcase Name maralixibat Downcase Name bile duct diseases Downcase Name digestive system diseases Downcase Name biliary tract diseases Downcase Name cholestasis Downcase Name pruritis

Browse Conditions

Sequence: 193793462 Sequence: 193793463 Sequence: 193793464 Sequence: 193793465 Sequence: 193793466 Sequence: 193793467 Sequence: 193793468 Sequence: 193793469 Sequence: 193793470 Sequence: 193793471 Sequence: 193793472 Sequence: 193793473 Sequence: 193793474 Sequence: 193793475 Sequence: 193793476 Sequence: 193793477 Sequence: 193793478 Sequence: 193793479 Sequence: 193793480
Mesh Term Alagille Syndrome Mesh Term Pruritus Mesh Term Syndrome Mesh Term Disease Mesh Term Pathologic Processes Mesh Term Skin Diseases Mesh Term Skin Manifestations Mesh Term Cholestasis, Intrahepatic Mesh Term Cholestasis Mesh Term Bile Duct Diseases Mesh Term Biliary Tract Diseases Mesh Term Digestive System Diseases Mesh Term Liver Diseases Mesh Term Heart Defects, Congenital Mesh Term Cardiovascular Abnormalities Mesh Term Cardiovascular Diseases Mesh Term Abnormalities, Multiple Mesh Term Congenital Abnormalities Mesh Term Genetic Diseases, Inborn
Downcase Mesh Term alagille syndrome Downcase Mesh Term pruritus Downcase Mesh Term syndrome Downcase Mesh Term disease Downcase Mesh Term pathologic processes Downcase Mesh Term skin diseases Downcase Mesh Term skin manifestations Downcase Mesh Term cholestasis, intrahepatic Downcase Mesh Term cholestasis Downcase Mesh Term bile duct diseases Downcase Mesh Term biliary tract diseases Downcase Mesh Term digestive system diseases Downcase Mesh Term liver diseases Downcase Mesh Term heart defects, congenital Downcase Mesh Term cardiovascular abnormalities Downcase Mesh Term cardiovascular diseases Downcase Mesh Term abnormalities, multiple Downcase Mesh Term congenital abnormalities Downcase Mesh Term genetic diseases, inborn
Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-list Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor Mesh Type mesh-ancestor


Sequence: 48394591 Sequence: 48394592
Agency Class INDUSTRY Agency Class INDUSTRY
Lead Or Collaborator lead Lead Or Collaborator collaborator
Name Mirum Pharmaceuticals, Inc. Name Clinigen, Inc.


Sequence: 30812160
Gender All
Minimum Age 12 Months
Maximum Age N/A
Criteria Inclusion Criteria: Informed consent and assent provided, as applicable, per Institutional Review Board (IRB) or Ethics Committee (EC) Clinical and/or genetic diagnosis of ALGS Male or female participants aged >12 months and ≥5 kg with clinically significant cholestatic pruritus associated with ALGS Willingness to adhere to an acceptable method of contraception until treatment discontinuation by females of childbearing potential who are sexually active or who become sexually active during the program No other therapeutic options or access to any ongoing ALGS clinical trials Exclusion Criteria: Diagnosis with a cholestatic liver disease other than ALGS Female who is pregnant or breastfeeding Clinically significant cardiovascular, hepatic, gastro-intestinal, pulmonary, neurologic, infectious or renal disease or other medical condition that, in the opinion of the physician or Medical Monitor, would preclude participation in the program Past medical history of compensated or decompensated cirrhosis Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of maralixibat or its mode of action
Adult True
Child True
Older Adult True

Calculated Values

Sequence: 254051726
Number Of Facilities 14
Registered In Calendar Year 2020
Were Results Reported False
Has Us Facility True
Has Single Facility False
Minimum Age Num 12
Minimum Age Unit Months


Sequence: 4394351 Sequence: 4394352 Sequence: 4394353 Sequence: 4394354
Url https://ghr.nlm.nih.gov/condition/alagille-syndrome Url https://clinicaltrials.gov/ct2/info/fdalinks Url https://mirumpharma.com Url https://algseap.com/
Description Genetics Home Reference – Alagille syndrome Description US FDA Resources Description Mirum Pharmaceuticals homepage Description Mirum webpage specific to ALGS EAP

Responsible Parties

Sequence: 28924524
Responsible Party Type Sponsor